Bortezomib in previously treated phosphatase and tension homology-deficient patients with advanced intrahepatic cholangiocarcinoma: An open-label, prospective and single-centre phase II trial

Abstract

• There is a limited strategy for the second-line option of intrahepatic cholangiocarcinoma (ICC).

• This investigator-initiated phase 2 study evaluated bortezomib in ICC patients with phosphatase and tension homology deficiency.

• The overall response rate was 18.75% and the overall survival was 7.2 months in the intent-to-treat cohort.

• These results justify further developing bortezomib in ICC patients with PTEN deficiency.

Keywords

bortezomib / intrahepatic cholangiocarcinoma / PTEN

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2024 The Authors. Clinical and Translational Medicine published by John Wiley & Sons Australia, Ltd on behalf of Shanghai Institute of Clinical Bioinformatics.